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Summarising - Assignment Example

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Researchers used two versions of genetically engineered virus of Adeno-Associated Virus. One that carried enzymes that cut DNA in an exact spot within living mice’s…
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Download file to see previous pages High and Sangamo BioSciences, Inc., scientists used Zinc Finger Nucleases (ZFNs) genetically engineered enzymes that act as molecular word processors to edit mutated sequences of DNA. Since scientists have learnt how to design custom-matched ZFNs to a specific gene location, they designed ZFNs specifically for factor nine genes (F9) and used it in conjunction with a DNA sequence to restore the normal gene function lost in hemophilia. ZFNs are more advantageous compared to conventional genes since they target a precise place in a sequence of chromosomes (High). Conventional genes may deliver a replacement gene to an unwanted location resulting to bypassing of normal biological regulatory components controlling the gene hence cause risk of “insertion mutagenesis”.
An inherited single-gene mutation in hemophilia impairs a patient’s ability to produce blood-clotting proteins. This results to spontaneous life-threatening bleeding episodes. Hemophilia has two major forms, A and B, which occurs solely in males. They result from lack of clotting factors VIII and IX respectively. In a recent study, researchers used genetic engineering to produce a hemophilia B mouse. Prior treatment, the mouse depicted no levels of clotting factor IX since previous studies by different researchers shown that ZFNs could accomplish genome editing if injected into mice for sickle cell disease treatment. However, the ex living animal approach was not feasible for many human genetic diseases affecting whole organ system (Shangzhen). Therefore, the current study was testing genome-editing effectiveness when performed in a vivo.
The researchers designed two vector versions using Adeno-associated virus. One vector ferried ZFNs for editing while the other delivered a correctly functioning version of the F9 gene. Since different mutations in the same gene may cause hemophilia, the process replaced seven different coding sequences, covering 95% of the ...Download file to see next pagesRead More
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